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A monocentric study of steroid-refractory acute graft-versus-host disease treatment with tacrolimus and mTOR inhibitor

Bone Marrow Transplantation volume 55, pages 86–92 (2020)Cite this article

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Abstract

Acute graft-versus-host disease (aGVHD) remains one of the leading causes of morbidity and mortality after allogeneic hematopoietic stem cell transplantation. No consensus exists on the best second-line treatment of steroid-refractory acute GVHD (SR-aGVHD). Previously published smaller studies on the use of sirolimus in SR-aGVHD treatment report a response rate of 57 to 86%, with 40% overall survival. The association of tacrolimus and mTOR inhibitor is supported by pre-clinical data and has been used as GVHD prophylaxis. We report 42 patients who received tacrolimus and mTOR inhibitor as a second- or third-line treatment of SR-aGVHD. Thirty-one patients were treated in second-line, with an overall response rate of 48.5% (complete response: 42%). Eleven patients were treated in third-line, with an overall response rate of 27%. Thirty-eight patients had at least one episode of infection, due to bacteria, viruses, fungi and parasites in 61, 42, 12 and two episodes, respectively. For patients treated in second-line, six-month and one-year survival were 61% and 42%, respectively. None of the patients treated in third-line survived. These results were not promising enough to initiate a phase three randomized clinical trial, but tacrolimus and mTOR inhibitor can be discussed among other options for patients with SR-aGVHD.

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Authors and Affiliations

  1. Service d’hématologie-greffe, Hôpital Saint-Louis, Université Paris Diderot, Paris, France

    Alienor Xhaard, Flore Sicre de Fontbrune, David Michonneau, Aurelien Sutra del Galy, Tereza Coman, Simona Pagliuca, Marie Robin, Regis Peffault de Latour & Gerard Socie

  2. Service de pharmacologie, Hôpital Européen Georges-Pompidou, Paris, France

    Manon Launay

  3. Service d’hématologie adolescents—jeunes adultes, Hôpital Saint-Louis, Université Paris Diderot, Paris, France

    Nathalie Dhedin

  4. Inserm UMR1160, Paris, France

    Gerard Socie

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  1. Alienor Xhaard
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  2. Manon Launay
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Correspondence to Alienor Xhaard.

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Xhaard, A., Launay, M., Sicre de Fontbrune, F. et al. A monocentric study of steroid-refractory acute graft-versus-host disease treatment with tacrolimus and mTOR inhibitor. Bone Marrow Transplant 55, 86–92 (2020). https://doi.org/10.1038/s41409-019-0633-y

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